Gahan J Pandina, Joan Busner, Lucas Kempf, Joan Fallon, Larry D Alphs, Maria T Acosta, Anna-Karin Berger, Simon Day, Judith Dunn, Victoria Villalta-Gil, Margaret C Grabb, Joseph P Horrigan, William Jacobson, Judith C Kando, Thomas A Macek, Manpreet K Singh, Arielle D Stanford, Silvia Zaragoza Domingo
The 1983 Orphan Drug Act in the United States (US) changed the landscape for development of therapeutics for rare or orphan diseases, which collectively affect approximately 300 million people worldwide, half of whom are children. The act has undoubtedly accelerated drug development for orphan diseases, with over 6,400 orphan drug applications submitted to the US Food and Drug Administration (FDA) from 1983 to 2023, including 350 drugs approved for over 420 indications. Drug development in this population is a global and collaborative endeavor...
2024: Innovations in Clinical Neuroscience